Project Title

A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, postpolycythemia vera or post-essential thrombocythemia myelofibrosis

Official Title

A phase 1/2a study to evaluate safety, pharmacokinetic and pharmacodynamic dose escalation and expansion study of PXS-5505 in patients with primary, postpolycythemia vera or post-essential thrombocythemia myelofibrosis

Project Summary

The study consists of two phases: a dose escalation phase and a cohort expansion phase. The dose escalation phase will follow a 3+3 design with a starting dose of 100 mg twice daily, and a treatment duration of 4 weeks. Patients will be able to participate in more than one dose level.

During the cohort expansion phase, 24 patients will be treated at the dose determined appropriate based on safety, pharmacokinetic and pharmacodynamic results from the dose escalation phase, for a period of up to 6 months. Patients from the dose escalation phase will be able to participate in the cohort expansion phase.

There will be no washout period between dose escalation and dose expansion cohorts.

Blood Disorder

  • Myelofibrosis

Patient Recruitment Details

Patient recruitment status: Open

Number of Patients (Globally)

24 participants.